Navigating the “Really Exciting” Specialty Pharmaceutical Pipeline

The specialty pharmaceutical landscape is rapidly evolving, with a robust pipeline of innovative therapies poised to transform the treatment of a wide range of conditions, according to experts in the space. From groundbreaking cell and gene therapies to novel agents targeting rare diseases and complex immunological disorders, managed care professionals must stay informed to effectively manage the growing specialty drug spend.

The specialty pharmaceutical pipeline is rapidly evolving, with a robust and diverse set of new therapies in development across various therapeutic areas. | Image credit: Karanov images - stock.adobe.com

At the AMCP Nexus 2024 conference, experts provided a comprehensive overview of the latest trends, recent approvals, and upcoming pipeline developments across key therapeutic categories, equipping decision-makers with the insights needed to navigate this dynamic and ever-changing space.

Overall Specialty Pipeline Trends

The specialty pharmaceutical pipeline is rapidly evolving, with a robust and diverse set of new therapies in development across various therapeutic areas. Key trends include the continued growth of cell and gene therapies, particularly for rare and genetic disorders, as well as innovative treatments for immunology, pulmonary, and central nervous system (CNS) conditions.

“When we look at the pipeline for biosimilars, in 2024 there have been 16 biosimilars and we anticipate at least 43 more over the course of the next 3 years, all of which contributes to the specialty pipeline,” said YuQian Liu, PharmD, senior director of specialty and clinical solutions, Prime Therapeutics.

Recent Specialty Drug Approvals

The dermatology space has seen several recent biosimilar approvals, which are playing an increasingly important role in expanding treatment options and improving affordability for patients. In the past quarter, the FDA approved several biosimilars for key dermatological conditions, including psoriasis and atopic dermatitis. Recent specialty approvals in dermatology include deuruxolitinib (Leqselvi), nemolizumab-ilto (Nemluvio), and lebrikizumab-lbkz (Ebglyss).

Other recent specialty approvals include:

• Palopegteriparatide (Yorvipath) for hypoparathyroidism in adults
• Seladelpar (Livdelzi) for primary biliary cholangitis
• Axatilimab-csfr (Niktimvo) for cGVHD
• Donanemab-azbt (Kisunla) for Alzheimer disease
• Arimoclomol (Miplyffa) for Niemann-Pick disease type C
• Levacetylleucine (Aqneursa) for Niemann-Pick disease type C

Recent biosimilar drug approvals include eculizumab-aagh (Epysqli), aflibercept-abzv (Enzeevu), and aflibercept-ayyh (Pavblu). Additionally, recent 505(b)(2) approvals include paliperidone palmitate (Erzofri) and bortezomib.

Endocrine Trends

Although the endocrine category is not typically considered a specialty therapeutic area, more expensive and self-administered products are now being included in this space. The forecast shows an increase in the pharmacy trend for the endocrine category, particularly for the commercial population, driven by both rising drug costs and changes in utilization. Key pipeline products in the endocrine space include octreotide, govorestat (AT-007), and crinecerfont, which are expected to have Prescription Drug User Fee Act (PDUFA) dates later this year. Additionally, the pipeline includes new treatments for conditions like Cushing disease and hypothyroidism, which are anticipated to contribute to the overall trend in this category.

Gastroenterology Trends

The gastroenterology space is seeing notable trends in both the medical and pharmacy benefits. On the medical side, the trends are remaining fairly flat, likely due to biosimilar competition keeping prices down, although utilization is increasing. On the pharmacy side, the trend is increasing, driven by the influx of new and innovative products coming to market for conditions like Crohn disease and ulcerative colitis. These new therapies are launching at higher costs compared with traditional treatments, contributing to the rising pharmacy per-member per-month (PMPM) trend in this category. Key pipeline products to watch include glepaglutide (ZP1848), a new treatment for short bowel syndrome with an orphan drug designation and a forecasted wholesale acquisition cost of $500,000 to $750,000 per year.

Immunology Trends

Some of the key pipeline drugs in immunology include new treatments for eosinophilic esophagitis, additional competition in the atopic dermatitis market, and a product targeting dermatological myositis, a condition that has historically lacked targeted therapies.

The presentation delved into the autoimmune category, which includes drugs like tumor necrosis factor (TNF) inhibitors and other biologics used to treat conditions such as rheumatoid arthritis, psoriasis, and ankylosing spondylitis. This rise in the pharmacy trend is primarily driven by the influx of new oral agents entering the market, which are launching at high price points and competing with existing injectable products.

Rare Disease Trends

The presentation highlighted the robust pipeline of treatments for rare diseases, a category that is expected to have a significant impact on managed care organizations. The rare disease trend forecast shows a substantial increase, particularly for the Medicare population, driven by the high cost of these specialized therapies. Many of the rare disease products in development are targeting genetic disorders where patients have historically had limited to no treatment options available.

For example, the pipeline includes elamipretide (MTP-131), the first potential treatment for Barth syndrome, a rare X-linked recessive disorder affecting male infants, with a forecasted wholesale acquisition cost (WAC) of $750,000 to $1 million per year.

Pulmonary Trends

The pulmonary category includes drugs for conditions such as asthma, chronic obstructive pulmonary disease (COPD), cystic fibrosis, and pulmonary hypertension. The forecast shows a decrease in the medical PMPM trend for this category, likely due to more patients shifting to the pharmacy side. However, the pharmacy PMPM trend, especially for Medicare, is expected to increase. Key drivers of this trend include deutivacaftor/tezacaftor (VX-121/REZ/VEX-561), a new cystic fibrosis product with a PDUFA date in January 2025, as well as IL-33 antagonist agents being studied for COPD, which could be a game-changer if they are able to break into that market.

Gene and Cell Therapies

The pipeline for cell and gene therapies is also robust, with 25 products in development across various therapeutic areas, including endocrine, metabolic, and nutritional disorders, as well as hematology and ophthalmology. These pipeline products represent the continued advancements in this rapidly evolving field and the potential to address unmet needs for patients with rare and genetic diseases.

One notable upcoming product is marstacimab-hncq (Hympavzi), which would be the first therapy that can be used to treat either hemophilia A or hemophilia B. It has a new mechanism of action as a tissue factor pathway inhibitor, representing a departure from the traditional factor replacement therapies.

CNS Trends

The pipeline for CNS disorders includes about 11 new entities, primarily focused on indications for Alzheimer disease, multiple sclerosis, and drug-based syndrome. The presentation highlighted 2 deep dives on Alzheimer disease treatments, including valitramiprosate (ALZ-801), an oral amyloid β protein inhibitor, and blacamesine (ANAVEX2-73), another oral agent that showed promising results in slowing cognitive decline, though it did not demonstrate statistically significant improvements in activities of daily living.

Cardiovascular Trends

The pipeline for the cardiovascular category includes about 6 therapies in phase 3 trials covering various indications. These pipeline products feature different routes of administration, including subcutaneous, nasal, and oral formulations. One notable pipeline candidate is olezarsen (AKCEA-APOCIII-LRx), an antisense oligonucleotide that inhibits the production of apolipoprotein C-III and has the potential to be the first FDA-approved therapy for familial chylomicronemia syndrome.

Biosimilar Trends

The presentation highlighted the growing importance of biosimilars in the specialty pharmaceutical landscape. The data presented showed significant decreases in the average sales price (ASP) of reference biologics since the launch of their biosimilar counterparts, ranging from 44% to 91% reductions. However more recent quarters have seen fluctuations in these ASP trends, driven by the influx of new biosimilar approvals and the implementation of various distribution models and provider discount strategies.

Looking ahead, the pipeline for biosimilars is robust, with multiple products expected to launch for high-volume biologics like adalimumab (Humira), infliximab (Remicade), and pegfilgrastim (Neulasta). The presentation also highlighted that some of these biosimilar categories, such as those for omalizumab (Xolair) and denosumab (Prolia/Xgev), will be the first to market, potentially disrupting the treatment landscape.

“If you take one thing away from this presentation, it's that the specialty space is actually really exciting,” said Soumya Vishwanath, PharmD, senior manager of formulary management, Prime Therapeutics. “There's a lot of new pipeline products for a lot of different indications that don't have treatments. For a lot of these patients this is the first time that these patients will be able to even get a treatment for their disorders. And for a lot of them, a lot of these new novel entities will provide a unique mechanism, or a unique way that delivers for these patients.”

Reference

Liu Y, Vishwanath S. Specialty pharmaceuticals in development. Presented at: AMCP Nexus 2024; October 14-17, 2024; Las Vegas, NV.